RARE Daily

Novartis Reports Positive Interim Analysis from Phase 2 Study in Rare Kidney Disease

June 7, 2021

Novartis reported positive new interim phase 2 data showing that its experimental therapy iptacopan stabilized kidney function in patients with C3 glomerulopathy.

Complement-driven renal diseases, which include C3 glomerulopathy (C3G), are thought to be partly caused by an overactivation of the alternative complement pathway—part of the immune system—creating an inflammatory response, which can lead to kidney damage. CDRDs mainly affect adolescents and young adults and can often lead to kidney failure which requires dialysis or transplantation and can lead to premature death. Approximately 50 percent of C3G patients progress to kidney failure within 10 years of diagnosis. Among patients who have undergone kidney transplantation, disease recurrence is not uncommon, with one study seeing an estimated 30 percent risk of transplant loss at five years and a 70 percent risk of transplant loss at 10 years.

Iptacopan is an investigational, first-in-class, orally administered factor B inhibitor of the alternative complement pathway, targeting one of the key drivers of these diseases. It has the potential to become the first targeted therapy to delay progression to dialysis in C3G. Discovered at the Novartis Institutes for BioMedical Research, iptacopan is currently in development for a number of complement-driven renal diseases where significant unmet needs exist, including C3G, IgA nephropathy, atypical hemolytic uremic syndrome, and membranous nephropathy, as well as the blood disorder PNH.

The new interim analysis data from the open-label phase 2 study in patients with C3G showed twice-daily 200mg iptacopan stabilized kidney function, as measured by change in estimated glomerular filtration rate slope, a key measure of kidney clearance function that estimates the rate of blood passing through and being filtered by the kidneys. The kidney function of 12 patients treated with iptacopan for 12 weeks was compared with their historical kidney function data for the two-year period before they entered the study (or since diagnosis was made where this was less than two years). Furthermore, seven C3G patients who received iptacopan for up to 25 weeks in a long-term extension study showed ongoing eGFR stabilization, suggesting extended iptacopan treatment may prolong the time to, or even potentially prevent, the development of kidney failure.

Author: Rare Daily Staff

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